Samuel was four years old when, in November 2012, he began to behave differently. He couldn't sit still, he lost weight and was thirsty all day. “He could drink a big bottle of water in one gulp. He drank so much that he started to wet the bed,” recalls his mother, María Rego. Two months later, the boy was hospitalized. After many sleepless nights and several visits to the pediatrician, a glucose test result of 400—between 70 and 120 is normal—revealed what was happening. “The child had type 1 diabetes. We finally understood everything, but it was still a blow, a moment that changes your life,” says this neighbor from A Pobra do Caramiñal (A Coruña) who works as a computer technician at the Xunta de Galicia.
Like Samuel, nearly 100,000 people live in Spain with this ailment, in which the body runs out of insulin because an erratic attack by the immune system destroys the beta cells that produce it in the pancreas. The patient is thus forced for life to adopt strict routines to control blood glucose levels and inject the right doses of this hormone. Type 1 diabetes, unlike type 2 – which develops at older ages, associated with obesity and a sedentary lifestyle – is usually diagnosed in childhood and adolescence.
For more than a century, since Frederick Banting discovered the hormone in 1921 and managed to turn a fatal disease into a chronic one, medicine has treated type 1 diabetes as a metabolic disease, in which the goal is to make up for the lack of insulin. with injections. Something that is beginning to change thanks to new treatments that seek to prevent the disease from developing. “We are taking the first steps towards type 1 diabetes without insulin. And this is a transcendental idea,” says Welshman Colin Dayan, a world-renowned figure in the disease who announced last Wednesday. inaugural conference of the XXXV Congress of the Spanish Diabetes Society in Granada.
Teplizumab, the active ingredient marketed under the brand name Tzield, is the name of the first of these therapies that has been shown to be safe and effective. “The drug prevents T lymphocytes from attacking beta cells and slows down the development of the disease for two or three years. This is relevant because it frees patients, who are usually children and adolescents, from taking insulin every day during this time. But even more important is that it can be the first step to delay type 1 diabetes until it can be done indefinitely,” explains Spanish immunologist Francisco León, whose research in the United States managed to demonstrate the safety and effectiveness of the drug.
Tzield was approved in the US in November 2022 and the European Medicines Agency (EMA) is now studying whether to do the same in the EU. During this time, the company with which León developed the drug—ProventionBio—has been purchased for more than 2.7 billion euros by the pharmaceutical giant Sanofi. “We are now evaluating whether teplizumab is also effective when diabetes has already started. The first results presented show that it can protect beta cells that are still functioning in the pancreas, which could improve the prognosis and reduce the need for insulin. But we need to better understand these promising results,” says José Luis Guallar, medical director of Sanofi in Spain.
This immunotherapy has been the first of all the strategies attempted by medicine to attack the causes of type 1 diabetes to have some success, highlights the president of the SED, Antonio Pérez. “For decades we have investigated what causes the immune system to attack beta cells. We know that there is a genetic predisposition, but only one in 10 people who have it will end up developing the disease. So there has to be something that sets the process in motion, but we still don't know what it is,” he declares.
One of the main hypotheses is that some viral infections trigger the excessive reaction of the immune system. Researchers from the Agency for Health Quality and Evaluation of Catalonia (AQUAS) and the Hospital del Mar (Barcelona) discovered that in this community the incidence of the disease skyrocketed during the pandemic, something that has also been observed in other countries, which indicates to coronavirus as a cause. With the money received from Sanofi, Francisco León has started another investigation into a virus called coxsackie B. “It is the only one that has shown a statistically significant association with type 1 diabetes in the two largest studies carried out, TEDDY and DIPP, with more of 600,000 subjects studied. This association has been observed in 15 countries,” he states.
The transplantation of pancreatic islets containing beta cells has been another bet that has so far failed. “It has been achieved, but the immune system destroys them again. To avoid this, we should subject the patient to immunosuppressive treatment equivalent to that of a person receiving a liver, for example. And this has risks. It is justified when there are no alternatives to save the patient's life, but not with diabetes that can be treated with insulin,” adds Pérez.
The new strategy that opens with teplizumab—along with nine other molecules under investigation—completely changes the rules of the game, Dayan celebrates. “With them, if all the trials end successfully, we can do selective immunosuppression, without the risk of serious infections. And adopt an approach that is already followed in other autoimmune diseases, such as rheumatoid arthritis: combine different drugs and adapt the necessary ones to the patient's evolution,” he says. This selective immunosuppression would also make pancreatic islet transplantation viable: “The new drugs would protect the transplanted beta cells in a safe way,” continues this expert.
The potential of teplizumab and the other treatments in development currently faces, however, an important obstacle: How do we know who needs to be treated? Medicine has the tools to know which people will develop type 1 diabetes. These are tests that detect antibodies in the blood that reveal that the process that will destroy beta cells has already begun, even if the patient does not yet suffer any symptoms. .
Samuel has a twin brother, Marcos, and a younger sister who is 12 years old. “Neither of them have had these tests done. Sometimes I wonder if it would be worth it, that maybe it is better to know beforehand what has to come, but the truth is that doctors are right when they say that there is no reason to do them and that they are not indicated. Why are you going to suffer if there is no treatment that can help them. If the EMA approves teplizumab, then there would be a reason to do them,” says his mother.
Immediate relatives of a patient with type 1 diabetes have 10 times the risk of developing the disease than the general population. “The logical thing would be to start screening with these people. The problem is that 90% of diagnoses occur in people without any family history, so this strategy would only allow us to detect a minimal part of the cases,” explains Pérez.
For Dayan, to overcome this problem it would be necessary to universalize screening in children with a first test at three or four years of age, which would allow up to 70% of potential cases to be detected. “It is very possible that it will be necessary to repeat it at eight or nine years of age to identify children in whom the autoimmune process has started later, although there is still a lot of debate about that. It is a simple test, which can be done with a finger prick, which costs about 20 euros and which could be managed by the child's doctor, as he already does with vaccines,” she defends.
The president of the SED, however, is more cautious. “There is no country that has that established. We must take into account the costs, the price of the treatment, the management of the analyzes and all the impact it would have on the health system. And to think that, if the responsibility falls on pediatricians, this would overload primary care even more,” says Pérez. A dozen scientific societies have created a working group in Spain to assess the best strategy to follow in the face of the possible approval of teplizumab by the EMA.
While they wait for medical advances, the patients' families are excited. “Living with type 1 diabetes at home is very complex. You have to measure everything with the child. What he does, what he eats, what he injects. Every day. The new intelligent devices that calculate glucose levels and administer the necessary insulin have been a great advance, but a scenario in which patients do not need it seems like a dream today,” concludes Rego, who also chairs the patient association of the disease in Galicia, ANEDIA.
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